Opportunities and Challenges of Real-World Data (RWD) in Benefit-Risk Assessment (BRA)

• Activity Number: 440 - Let’s Make Everyone and Everything Count! Benefit-Risk Assessment Challenges, Lessons and Impacts in the Age of Big Data from Clinical Trials to Real-World Evidence
• Type: Topic Contributed
• Date/Time: Thursday, August 6, 2020 : 10:00 AM to 11:50 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Emilie Scherrer*
• Companies: Merck & Co Inc
• Keywords: real world data; benefit risk assessment; genomics data; genomic data; health technology assessment; linking data

Abstract

RWD allows us to increase external validity of BRA. Patient groups excluded from clinical trials, such as the elderly or those with comorbidities, may not be represented in traditional BRA. Health technology assessment (HTA) is becoming increasingly important throughout the world and often requires specific data local to the market. Clinical trial data may not accurately represent the patient population or treatment landscape within a specific market where the BRA is required. Therefore, RWD can help add to BRA to be tailored to a specific system. For instance, the prevalence of melanoma subtypes differ in Asian ethnicities compared to European, and diagnosis rates depend on what guidelines and national screening programs are in place. Genomic data can add an additional layer of value for conducting BRA, allowing for additional subgroup analyses beyond basic patient baseline and clinical characteristics (e.g. age, ethnicity, disease stage). Our study will explore such opportunities of linking real world clinical and genomic data to better inform treatment decisions. Incorporating RWD into BRA allows results to be more representative of patients treated in clinical practice.